What the trial was testing
The ROAR enrolled 206 patients with biliary tract cancer. The study was sponsored by Novartis Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
About 50% tumor response rate in BRAF-mutant biliary tract cancer.
The Lancet Oncology · 2020 · NCT02034110
These findings — that in advanced BRAF-mutant biliary tract cancer on dabrafenib plus trametinib — were published in the The Lancet Oncology and represent the headline result of the study.
Researchers tracked outcomes across 206 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with biliary tract cancer, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Dabrafenib (Tafinlar) plus trametinib (Mekinist) is FDA-approved for any solid tumor with a BRAF V600E mutation, including biliary tract cancer, and available now. Ask an oncologist about BRAF testing on your tumor — most centers test routinely for advanced bile duct cancer.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open biliary tract cancer trials
Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of BL-M05D1 in Subjects With Solid Tumors
The objective of this study is to evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of BL-M05D1 in Subjects with Advanced or Metastatic Solid Tumors.
On-treatment Surveillance of Tumor Evolution and Response to Systemic Treatment in Bile Duct and Liver Cancer
The BILLIONSTARS study is a prospective, single arm observational study inviting patients diagnosed with hepatocellular carcinoma (HCC) or cholangiocarcinoma (CCC) who are to be recommended locoregional intervention by surgery, ablation or transarterial chemoembolization and/or antitumoral medical treatment. The aim is to investigate how tumor- and individual-related factors affect response to treatment. To this end, circulating tumor DNA, immune cells and various proteins will be analyzed in repeated blood samples taken before, during and after completion of systemic treatment. When applicable, analyses will also be performed on tumor tissue from resected tumors and biopsies, and in some cases also from autopsies